Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT ID: NCT05145062

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

8 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-12-21

Study Completion Date

2038-07-14

Brief Summary

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Primary Objectives:

Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT)

Secondary Objectives:

* Long-term efficacy of the biological treatment effect of BIVV003 in SCD
* Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events
* Long-term efficacy of the biological treatment effect of ST-400 in TDT
* Long-term efficacy of the clinical treatment effect of ST-400 in TDT

Detailed Description

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The total study duration is up to 15 years of follow-up post BIVV003 and/or ST-400 infusion.

Conditions

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Blood and Lymphatic Diseases

Keywords

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Sickle Cell Disease (SCD), Beta-thalassemia (TDT)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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BIVV003 Cohort

All participants treated in parent and future studies with BIVV003

BIVV003

Intervention Type DRUG

Solution for intravenous administration

ST-400 Cohort

All participants treated in parent studies with ST-400

ST-400

Intervention Type DRUG

Solution for intravenous administration

Interventions

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BIVV003

Solution for intravenous administration

Intervention Type DRUG

ST-400

Solution for intravenous administration

Intervention Type DRUG

Other Intervention Names

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SAR445136

Eligibility Criteria

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Inclusion Criteria

* Received treatment with BIVV003 or ST-400 in one of the parent studies (ACT16222, ST- 400-01) or any future studies with BIVV003
* Capable of giving signed informed consent (and if applicable assent)

Exclusion Criteria

* Unable to comply with study visit schedule or study procedures
* Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the participant unsuitable for participation in the study The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Minimum Eligible Age

18 Years

Maximum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sangamo Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Monitor

Role: STUDY_DIRECTOR

Sangamo Therapeutics

Locations

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UCSF Benioff Children's Hospital

Oakland, California, United States

Site Status

University of California Davis Health System

Sacramento, California, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Henry Ford Health System

Detroit, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Other Identifiers

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LTS16653

Identifier Type: -

Identifier Source: org_study_id